@KGI April 4-5: “Huntington’s Disease: From Basic Science to Therapy”

Slated for Thursday and Friday, April 4-5, KGI’s third annual research symposium, “Huntington’s Disease: From Basic Science to Therapy” will be the most comprehensive research conference ever held at the institute. It will bring together many innovative thinkers from industry and academia with a goal of fast-tracking cutting-edge research into new treatment options for those affected by Huntington's Disease (HD), a hereditary neurodegenerative disorder.

Often-termed a “common” rare disease, more than 30,000 people in the United States have been diagnosed with HD and another 200,000 are at-risk for developing the disease. “One of the most well-known HD victims, the folk singer Woody Guthrie, died from HD-related complications in 1967 at the age of 55. He struggled for more than a decade with the increasingly severe symptoms of the disease” said event organizer Dr. Angelika Niemz. “We thought a lot about him when we were putting together this conference. We really wanted to emphasize how HD impacts individuals and their families, and particularly how it can destroy human creative potential.”

The two-day event will feature twelve talks and three panel discussions on translating discoveries into therapies, neurophysiology and brain imaging, and on experimental model systems. Speakers include Lisa Ellerby, associate professor at the Buck Institute for Research on Aging who will give a talk on “modeling Huntington’s Disease with induced pluripotent stem cells”, and Walter Koroshetz, deputy director of the National Institute of Neurological Disorders and Stroke, NIH, who will give a talk describing the “National Institutes of Health's role in therapy development for HD”, among others.

The symposium will also provide an opportunity for the outside community to learn more about the work of KGI research scientists and students related to HD. For example, research scientist Dr. Biranchi Patra and PhD student Michael de La Cruz, in the laboratory of Dr. Animesh Ray, are using synthetic biology techniques to engineer a site-specific DNA cleaving enzyme and a synthetic DNA piece to edit the human Huntington’s Disease gene in live human cells. The idea is to develop a rapid technology for correcting the HD gene mutation in human stem cells. If successful, this should enable a better understanding of the molecular mechanisms of HD and advance the possibility of a stem cell therapy against the currently incurable disease.

“KGI is committed to translational research that can benefit patients with rare, neglected, and intractable diseases. This conference will bring together people involved in all critical aspects of the research and development process for new HD therapies. We hope that by creating that type of interaction and energy, we can facilitate the beginnings of real breakthroughs for patients affected by HD, and their families,” Dr. Niemz said.